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1. 发明授权

US08895309B2 Myostatin inhibition for enhancing muscle and/or improving muscle function 有权
标题翻译：肌抑制素抑制增强肌肉和/或改善肌肉功能
公开(公告)号：US08895309B2
公开(公告)日：2014-11-25
申请号：US12516995
申请日：2007-11-29
申请人： Brian K. Kaspar , Jerrry R. Mendell
发明人： Brian K. Kaspar , Jerrry R. Mendell
IPC分类号： A61P21/00 , A61K35/76 , C07K14/015
CPC分类号： A61K48/00 , A61K9/0019 , A61K35/761 , A61K38/00 , A61K38/18 , C07K14/4703 , C12N7/00 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12N2799/025
摘要： The present invention relates to methods for inhibiting myostatin, a regulator of muscle mass, for muscle enhancement (including inducing hypertrophy and/or hyperplasia) as well as improving muscle function (including decreasing atrophy and/or increasing endurance, force and/or strength). Some of the methods involve delivering genes to cells using viral vectors such as a recombinant Adeno-associated virus (rAAV), lentivirus or equine-associated virus, or using other delivery techniques known in the art in order to inhibit myostatin. Examples of genes to be delivered are genes encoding proteins such as Follistatin, Follistatin-related gene-1 (FLRG-1), growth differentiation factor associated protein-1 (GASP-1) and myostatin precursor propeptide. In other methods of the invention, expression of proteins such as activin IIb and myostatin is inhibited by oligonucleotide techniques to effect muscle enhancement. All the methods have applications in the treatment of musculoskeletal and neurodegenerative disorders among others, as well as enhancing muscle in livestock.
摘要翻译：本发明涉及抑制肌肉生长调节剂肌肉增强（包括诱导肥大和/或增生）以及改善肌肉功能（包括减少萎缩和/或增加耐力，力和/或强度）的肌生成抑制素的方法，。一些方法涉及使用病毒载体如重组腺相关病毒（rAAV），慢病毒或马相关病毒或使用本领域已知的其它递送技术将基因递送至细胞以抑制肌生成抑制素。要传递的基因的实例是编码蛋白如Follistatin，Follistatin相关基因-1（FLRG-1），生长分化因子相关蛋白-1（GASP-1）和肌生成抑制素前体前肽的基因。在本发明的其它方法中，蛋白质如激活素IIb和肌生成抑制素的表达被寡核苷酸技术抑制以实现肌肉增强。所有方法都可用于治疗肌肉骨骼和神经退行性疾病，以及增强家畜的肌肉。

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